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- W4380204395 abstract "Children with Down syndrome have a 30% risk of developing a condition called transient abnormal myelopoiesis (TAM) during prenatal development. TAM is a preleukemic disorder that can progress to myeloid leukemia (ML-DS). Progression prevention from TAM to ML-DS was so far unsuccessful, while ML-DS treatment options are limited by the availability of clinical trials for Down syndrome patients. As groundwork for overcoming these issues, we use hematopoietic cells at appropriate development to put them into an aggressive TAM-like state. We used this model in combination with CRISPR-Cas9 technology to screen genes associated with FDA approved therapeutics. Results were incorporated with screening data from a ML-DS a cell line, therefore leading to the identification of potential gene targets for TAM and ML-DS. The potential of this approach is not only the identification of gene targets but also the discovery of associated therapeutics. In vitro testing has yielded promising results with the potential to add to the limited ML-DS treatment repertoire and to contribute to the long-term goal of preventing cancer in children with a premalignant state." @default.
- W4380204395 created "2023-06-11" @default.
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- W4380204395 date "2023-05-01" @default.
- W4380204395 modified "2023-09-27" @default.
- W4380204395 title "Identifying Gene Targets for Drug repurposing to prevent Cancer in children with a premalignant state" @default.
- W4380204395 doi "https://doi.org/10.1055/s-0043-1768543" @default.
- W4380204395 hasPublicationYear "2023" @default.
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